Myotonic Dystrophy Type 1 (DM1)NCT04835298
Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1
An observational study of Myotonic Dystrophy Type 1 (DM1), run by CHU de Reims at 1 site in France.
Trial summary
Myotonic dystrophy type 1 (DM1) is one of the most common neuromuscular diseases in adults. As respiratory dysfunction is the most common cause of death in patients with DM1, a respiratory disease progression must be monitored combining symptom screening and respiratory function testing, in order to identify the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy Type 1 (DM1)
- What is being tested?
- Dyspnea
- How many people
- 34 participants
- Where
- 1 siteFrance
- Study dates
- 3 Jul 2020to 14 Jun 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: adult patients with myotonic dystrophy type 1
Who may be able to join
- patient with myotonic dystrophy type 1 confirmed by genetic analysis
- with an age older than 18 years
Who cannot join
- an ongoing or recent (i.e. within the last 4 weeks prior to study recruitment) medical condition, including pulmonary exacerbations
- patient already under non-invasive mechanical ventilation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Characters) Dyspnea Assessment in Adult Patients With Myotonic Dystrophy Type 1: a Monocentric Pilot Study
Myotonic dystrophy type 1 (DM1) is one of the most common neuromuscular diseases in adults. As respiratory dysfunction is the most common cause of death in patients with DM1, a respiratory disease progression must be monitored combining symptom screening and respiratory function testing, in order to identify the appropriate time to initiate non invasive ventilation (NIV).
Dyspnea, one of the main respiratory symptoms, has been little studied in patients with DM1.
Read more
The main objective of this study is to provide the first multidimensional description of dyspnea in patients with DM1.
The secondary objectives are:
- To compare respiratory symptoms according to the presence or not of criteria from respiratory function testing to initiate NIV
- To assess associations between dyspnea and respiratory function testing
- To assess associations between dyspnea and number of Cytosine Thymine Guanine (CTG) repeats
- To assess associations between dyspnea and muscular strength
- To assess associations between dyspnea and BMI
- To assess associations between dyspnea and anxiety or depression
- To assess associations between dyspnea and cognitive impairment
- To assess associations between dyspnea and quality of life.
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Being tested
- Dyspnea
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DyspneaOther
questionnaires
| Study group | Role | What they receive |
|---|---|---|
| Myotonic dystrophy type 1 | adult patients with myotonic dystrophy type 1 Receives: Other: Dyspnea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dyspnea
Borg scale at rest and after 6 minute walking test
Measured: Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2021
- Study start3 Jul 2020
- Primary completion20 Jul 2021
- Study completion14 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy Type 1 (DM1)Studies of the same condition, open and under way.
- More trials run by CHU de ReimsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHU de Reims
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04835298
- Sponsor's protocol ID
- PO20065*
- Status last verified
- Jun 2022
- First submitted
- 31 Mar 2021
- Last updated
- 15 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characters) Dyspnea Assessment in Adult Patients With Myotonic Dystrophy Type 1: a Monocentric Pilot Study
- ClinicalTrials.gov ID
- NCT04835298
- Acronym / sponsor ID
- PO20065*
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- CHU de ReimsUniversity, hospital or other
- Study start
- 3 Jul 2020 (actual)
- Primary completion
- 20 Jul 2021 (actual)
- Study completion
- 14 Jun 2022 (actual)
- First posted
- 8 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Myotonic dystrophy type 1 (DM1) is one of the most common neuromuscular diseases in adults. As respiratory dysfunction is the most common cause of death in patients with DM1, a respiratory disease progression must be monitored combining symptom screening and respiratory function testing, in order to identify the appropriate time to initiate non invasive ventilation (NIV).
Dyspnea, one of the main respiratory symptoms, has been little studied in patients with DM1.
The main objective of this study is to provide the first multidimensional description of dyspnea in patients with DM1.
The secondary objectives are:
- To compare respiratory symptoms according to the presence or not of criteria from respiratory function testing to initiate NIV
- To assess associations between dyspnea and respiratory function testing
- To assess associations between dyspnea and number of Cytosine Thymine Guanine (CTG) repeats
- To assess associations between dyspnea and muscular strength
- To assess associations between dyspnea and BMI
- To assess associations between dyspnea and anxiety or depression
- To assess associations between dyspnea and cognitive impairment
- To assess associations between dyspnea and quality of life.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Myotonic dystrophy type 1 | – | Other: Dyspneaadult patients with myotonic dystrophy type 1 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- adult patients with myotonic dystrophy type 1
Key inclusion criteria (2 listed)
- patient with myotonic dystrophy type 1 confirmed by genetic analysis
- with an age older than 18 years
Key exclusion criteria (2 listed)
- an ongoing or recent (i.e. within the last 4 weeks prior to study recruitment) medical condition, including pulmonary exacerbations
- patient already under non-invasive mechanical ventilation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dyspneaquestionnaires | Other | Myotonic dystrophy type 1 |
Primary outcome
Dyspnea
Time frame: Month 6
Borg scale at rest and after 6 minute walking test
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myotonic Dystrophy Type 1 (DM1), run by CHU de Reims at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dyspnea (time frame: Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2022.